2 results
Not approvedWill not start
Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…
Approved WMORecruiting
Primary Objective: 1) To evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecundary Objective:1) To evaluate the efficacy of DTX301 in 3 response categories2) To evaluate the effect of…